A Novel TBI Free Conditioning Protocol for Haploidentical Transplant in Acquired Aplastic Anemia:

• Status: Recruiting
• Trial end date: 2021-06-30
• Target enrollment: 0
• Participant gender: All

Summary

Severe and very severe aplastic anemia are life threatening disorders for which allogeneic stem cell transplant is only curative treatment. However, matched sibling donor (MSD) is available in only 25-35% cases. Pakistan has a population of around 203 million but there is no donor registry available so there is no option available for matched unrelated donor (MUD) transplants . Haploidentical transplant represents only curative option for patients lacking MSD. Protocols involving post transplant cyclophosphamide require Total body irradiation (TBI) and utilize peripheral blood stem cell(PBSC) as graft source. TBI is not available in most of transplant centres across Pakistan due to lack of availability , cost and lack of expertise. The investigators have conceived a novel TBI free conditioning regimen to be used for haplo-identical Hemtopoeitic stem cell transplant in acquired aplastic anemia patients

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Armed Forces Bone Marrow Transplant Center, Rawalpindi, Pakistan; National Institute of Blood and Marrow Transplant (NIBMT), Pakistan
• Treatments: Cyclophosphamide

Criteria

Inclusion Criteria:

• Age >2 years and < 60 years

• Karnofsky performance status >= 70%

• Aplastic Anemia that meets the following criteria:

i. Peripheral Blood (must fulfill 2 of 3): ii. <500 neutrophils iii. <20,000 platelets iv. absolute reticulocyte count <40,000/microL

• Bone Marrow (must be ): markedly hypocellular (<25% of normal cellularity) with absence of reticulin and abnormal infiltrate

Exclusion Criteria:

• Presence of donor specific antibodies

• Fanconi anemia

• Cytogenetic abnormalities suggestive of myelodysplastic syndrome

• Prior HSCT

• Human immunodeficiency virus infection

• Active Hepatitis B virus infection

• Active /uncontrolled bacterial, viral , fungal infection or Tuberculosis

• Psychiatric illness

• Poor cardiac function (ejection fraction <40%)

• Poor pulmonary function (Forced vital capacity <50% predicted)

• Poor liver function (bilirubin >= 2mg/dL)

• Poor renal function (creatinine >= 2.0mg/dL or creatinine clearance <40)